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SYNGAP antisense oligonucleotide as therapy for human cognitive disorders

Unmet Need: Recent human genetic studies have suggested that mutations in the SYNGAP1 gene are linked to intellectual disability (ID), autism spectrum disorders (ASD), neurodevelopmental disorders, high rates of epilepsy, and schizophrenia. SYNGAP1 is a gene that encodes SynGAP, a GTPase-activating protein that is highly enriched in nerve cells in the...

Published: 5/9/2024 | Inventor(s): Richard Huganir, Ingie Hong, Yoichi Araki

Keywords(s): Biologics, CNS and Neurological Disorders, Cognitive Impairment, Disease Indication, Gene Therapy, Nucleic Acid, Single, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Psychiatry > Autism Spectrum Disorder, Clinical and Disease Specializations > Psychiatry > ADHD, Clinical and Disease Specializations > Psychiatry > Mood Disorders, Technology Classifications > Therapeutic Modalities > Gene Therapies

Method for using alternative splicing to control specificity of gene therapy

Unmet NeedViral-based gene therapy holds tremendous promise for the treatment of human diseases. Using genetically engineered carriers called vectors to deliver genes into cells, gene therapy can help replace a mutated gene with a healthy copy or even introduce a new gene into the body to fight a disease. However, an important concern when designing...

Published: 5/9/2024 | Inventor(s): Jonathan Ling, Seth Blackshaw

Keywords(s): Drug Delivery Mechanism, Gene Therapy, Single, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Research Tools > Vectors & Plasmids

Hexosamine Analogs for Regenerative Medicine and Diseases Associated with the Musculoskeletal System

Unmet Need:Osteoarthritis (OA) is the most common form of arthritis, affecting an estimated 32.5 million adults in the United States, with an increasing incidence as the population ages (see CDC) . OA is associated with considerable morbidity as a consequence of joint damage, resulting in pain, loss of joint range of motion, and disability. The disease...

Published: 5/9/2024 | Inventor(s): Kevin Yarema, Jennifer Elisseeff, Jeannine Coburn, Udayanath Aich

Keywords(s): Antagonists/Inhibitors, Arthritis, Biologics, Carbohydrates, Disease Indication, Drug Delivery Mechanism, Drug Delivery Vehicle, Joint Disorders, Novel, Osteoarthritis, Predicted Novelty, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Targets, Clinical and Disease Specializations > Rheumatology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Rheumatology > Arthritis, Clinical and Disease Specializations > Rheumatology > Osteoarthritis

Bioreducible Poly(beta-amino ester)s for siRNA Delivery

C12218: Efficient Delivery Vehicle for Controlled Gene Knockdown Novelty: This technology comprises the use of bioreducible polymer based nanoparticle system that can release siRNA cargo to the cytoplasm of cells to knockdown target gene expression in tissues. Value Proposition: Gene therapy is a developing medical field and there is a need for...

Published: 5/9/2024 | Inventor(s): Kristen Kozielski, Stephany Tzeng, Jordan Green

Keywords(s): Biologics, Brain Cancer, Cancers, Cell Therapy, Disease Indication, Drug Delivery Vehicle, Glioma, Nucleic Acid, Polymers, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Cell Therapies, Technology Classifications > Therapeutic Modalities > Dendrimers & Polymers, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Brain Cancer, Clinical and Disease Specializations > Oncology > Glioma

N-Hydroxysulfonamide Derivatives as New Physiologically Useful Nitroxyl Donors

Technology OverviewThe invention relates to N-hydroxysulfonamide derivatives that donate nitroxyl (HNO) under physiological conditions and are useful in treating and/or preventing the onset and/or development of diseases or conditions that are responsive to nitroxyl therapy, including heart failure and ischemia/reperfusion injury. Novel N-hydroxysulfonamide...

Published: 5/9/2024 | Inventor(s): Andrew Cohen, Frederick Brookfield, Vincent Kalish, Stephen Courtney, Lisa Frost, John Toscano

Keywords(s): Cardiovascular Diseases, Congestive Heart Failure, Congestive Heart Failure (CHF), Disease Indication, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Small Molecules, Clinical and Disease Specializations > Cardiovascular, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Cardiovascular > Congestive Heart Failure

N-Substituted Hydroxylamine Derivatives with Carbon-Based Leaving Groups as Physiologically Useful Nitroxyl (HNO) Donors

Technology OverviewThe disclosed subject matter provides certain N-substituted hydroxylamine derivative compounds, pharmaceutical compositions and kits comprising such compounds, and methods of using such compounds or pharmaceutical compositions. In particular, the disclosed subject matter provides methods of using such compounds or pharmaceutical compositions...

Published: 5/9/2024 | Inventor(s): Daryl Guthrie, John Toscano

Keywords(s): Cardiovascular Diseases, Disease Indication, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutic Substance Synthesis Method, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Cardiovascular, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities

Radiolabeled Thapsigargin Analogs Incorporated into Protease Activated Prodrugs

Various embodiments of this invention relate generally to targeted activation and delivery of therapeutic drugs to cells that produce prostate specific membrane antigen (PSMA), prostate specific antigen (PSA) or human glandular kallikrein 2 (hK2). Various embodiments relate more specifically to PSA, hK2 or PSMA-specific peptide prodrugs that become...

Published: 5/9/2024 | Inventor(s): Soren Christensen, John Isaacs, Samuel Denmeade

Keywords(s): Biologics, Cancers, Disease Indication, Peptide, Prostate Cancer, Single, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Men's Health, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Peptides, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Prostate Cancer

Nanoparticle Modification of Human Adipose-derived Mesenchymal Stem Cells for Brain Cancer and Other Neurological Diseases

Human Adipose-derived Mesenchymal Stem Cells can be engineered to synthesize and release anti-tumor proteins and therefore can be used as “Trojan Horses”. The inventors have created a novel technology which encompasses a solution containing biodegradable, polymeric nanoparticles that is combined with Freshly-extracted Adipose Tissue (F.A.T.) (from...

Published: 5/9/2024 | Inventor(s): Jordan Green, Alfredo Quinones-Hinojosa

Keywords(s): Biologics, Brain Cancer, Cancers, Cell Therapy, CNS and Neurological Disorders, Disease Indication, Drug Delivery Vehicle, Glioma, Nanoparticles, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type, Whole cell

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities > Cell Therapies, Technology Classifications > Therapeutic Modalities > Biologics, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Oncology > Brain Cancer, Clinical and Disease Specializations > Oncology > Glioma

Lipid Nanoparticles as Oral Vehicles of Immunotherapy

Unmet NeedType 1 diabetes (T1D) affects 1.25 million Americans currently and by 2050, this number is expected to reach 5 million. Though a currently untreatable autoimmune disease, one of the more promising approaches has been a costimulatory blockade of malfunctioning immune cells via administration of CTLA4-Ig. However, its efficacy is impeded by...

Published: 5/9/2024 | Inventor(s): Giorgio Raimondi, Julia Patrone, Xiomara Calderon-Colon, Olivia Tiburzi

Keywords(s): Diabetes, Disease Indication, Drug Delivery Mechanism, Drug Delivery Vehicle, Immunotherapy, Metabolic Disorders, Nanoparticles, Oral Administration, Single, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Diabetes > Type 1 Diabetes, Clinical and Disease Specializations > Diabetes, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms

Method to Enhance Pulmonary Dendritic Cell-mediated DNA Vaccination

Unmet NeedWith lung diseases, namely influenza and lung cancer, on the rise, pulmonary vaccination is a promising approach to elicit strong immunity to inhaled pathogens and lung cancer compared to systemic immunization strategies. In particular, inhaled DNA vaccination, a rapidly developing field, can potentially promote humoral and cellular immunity...

Published: 5/9/2024 | Inventor(s): Jung Soo Suk, Yoo Chun Kim, Justin Hanes

Keywords(s): Biologics, Cancers, Disease Indication, Drug Delivery Vehicle, Immunotherapy, Infectious Diseases, Influenza, Lung Cancer, Nanoparticles, Single, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type, Tuberculosis (TB), Vaccine

Category(s): Clinical and Disease Specializations > Infectious Diseases > Tuberculosis (TB), Clinical and Disease Specializations > Infectious Diseases > Influenza, Clinical and Disease Specializations > Immunology, Clinical and Disease Specializations > Immuno-Oncology, Clinical and Disease Specializations > Oncology > Lung Cancer, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Vaccines

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